Generic name |
sapropterin dihydrochloride |
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Strength/Form |
100 mg tablet 100 mg and 500 mg sachet with powder for oral solution |
Special Authority criteria |
Approval period |
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Initial coverage For the treatment of hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-responsive (BH4-responsive) phenylketonuria (PKU) in patients who meet ALL of the following initiation criteria: 1. a confirmed diagnosis of phenylketonuria (PKU) AND managed by a physician specialized in metabolic/biochemical diseases 2. adherence to low-protein diet and formulas AND baseline protein intake assessment by a dietitian 3. baseline blood phenylalanine (Phe) levels > 360 µmol/L despite adherence to a low-protein diet (requires at least 2 baseline levels during the preceding 3- to 6-month time frame) 4. ability to adhere to medication regimen |
6 months |
Renewal coverage For the treatment of HPA due to BH4-responsive PKU in patients who meet ALL of the following renewal criteria:
|
1 year |
Pregnant patients may qualify for coverage upon meeting the following modified criteria: 1. diagnosis of phenylketonuria (PKU) confirmed through an approved test and managed by a physician specialized in metabolic/biochemical diseases. 2. baseline blood phenylalanine (Phe) levels > 360 µmol/L despite adherence with all recommendations for dietary intervention and monitoring. Pregnant patients only require demonstration of one baseline Phe level > 360 µmol/L despite compliance with low-protein diet. Note: Pregnant patients will be eligible for funding for the duration of pregnancy. Renewal of funding will require meeting the criteria for non-pregnant patients. |
1 year |